Niemann-Pick Disease

What you'll learn

Course overview

Niemann Pick Disease (NPD) is a group of rare, inherited lysosomal storage disorders characterized by defective sphingomyelin metabolism, leading to lipid accumulation in organs such as the liver, spleen, lungs, and central nervous system. Subtypes include Type A, Type B, and Type C, each with distinct clinical features and prognoses. Early recognition is crucial, as progressive organ dysfunction and neurologic decline can result in severe disability or death. This course provides healthcare professionals with an in-depth review of NPD, including pathophysiology, clinical features, diagnostic strategies, pharmacologic interventions, and long term management.